Kevin C Kemp, Kelly Hares, Juliana Redondo, Amelia J Cook, Harry R Haynes, Bronwen R Burton, Mark A Pook, Claire M Rice, Neil J Scolding and Alastair Wilkins; Annals of Neurology, Accepted manuscript online: 13 MAR 2018 DOI: 10.1002/ana.25207
Our data provide proof-of-concept of gene replacement therapy, via allogeneic bone marrow transplantation, that reverses neurological features of Friedreich's ataxia with the potential for rapid clinical translation.
Tuesday, March 13, 2018
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